EMST: Expiratory Muscle Training for Persons With Neurodegenerative Disease

• Sponsor: VA Office of Research and Development (U.S. Fed)
• Overall Status: Completed
• CT.gov ID: NCT00856518
• Collaborator: University of Florida (Other)
• Enrollment: 42
• Location: 1
• Arms: 2
• Duration (Months): 65
• Patients Per Site Per Month: 0.6

Study Details

Study Description

Brief Summary

Respiratory difficulty is one of the primary factors leading to death in patients with Parkinson's Disease (PD) and Multiple Sclerosis. Both diseases are progressive degenerating diseases that cause difficulties in breathing, airway protection and swallowing. Patients with PD and MS typically become sedentary and lose endurance, maximal fitness levels and overall pulmonary function. Much of the research focus has been on the motor symptoms of PD and MS yet the pulmonary and swallowing complications are perhaps ultimately the most important disability as the diseases progress. The inability to generate adequate respiratory pressure is responsible for reduced cough magnitudes and cough response times. Cough is critical for the clearance of foreign materials in the airway helping to reduce infiltration of bacteria and subsequent respiratory infection. With reduced cough function an increased risk for pulmonary disease occurs due to a reduced ability to protect the airways. There are a number of promising outcomes from an expiratory strength-training program. By increasing expiratory muscle strength and expiratory pressure generation, effective breathing, clearance of the airway, and improved swallowing can occur. These explicit outcomes are predicted based on our experience with the use of an innovative device-driven, home-based expiratory strength training program focused on the expiratory muscles of respiration. This project focuses on following patients with PD and MS for an initial 5 weeks of strength training and them testing the outcome of a caregiver program for maintaining treatment effects.

Condition or Disease	Intervention/Treatment	Phase
Parkinson's Disease; Multiple Sclerosis	Device: EMST; Device: Sham	Phase 2/Phase 3

Detailed Description

The proposed investigation will:

Determine if 5 weeks of Expiratory Muscle Strength Training (EMST) increases maximal expiratory driving pressure (MEP) and improves swallow, cough and breathing function in individuals with PD and MS. Following the post assessment of the 5 week EMST program we will then evaluate three different modules for monitoring the continuation of the treatment while assessing patient quality of life and caregiver burden/satisfaction. This will help us determine if one particular home training method results in different physiological and functional outcomes.

Aim 1. Determine the effects of an EMST program on swallow function, voluntary cough production and breathing function in individuals with PD and MS identified as below normal limits for their age and sex (via physiological measures).

Hypothesis 1: There will significant and positive treatment effects following 5 weeks of EMST on the measures of swallow, cough production and breathing function in those with PD and MS following 5 weeks of treatment.

Aim 2: Determine the outcome of three uniquely structured home treatment monitoring programs in maintaining the EMST post treatment effect for patients with MS and PD. These programs are referred to as: Education Module (A), Question Only (B), and Education Module plus Question (C). The monitoring system will be provided by VitelNet, a leading provider of home health monitoring, clinician-based telemedicine Hypothesis 2: Program C will provide greater maintenance of the EMST treatment effect for both patient groups compared to programs A and B.

Aim 3: Determine the effects of the home monitoring programs for improving patient quality of life and caregiver burden/satisfaction.

Hypothesis 3: Program C will provide greater improvements in patient quality of life and caregiver burden compared to programs A and B.

Study Design

• Study Type: Interventional
• Actual Enrollment: 42 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: Expiratory Muscle Training for Persons With Neurodegenerative Disease
• Study Start Date: Mar 1, 2009
• Actual Primary Completion Date: Aug 1, 2014
• Actual Study Completion Date: Aug 1, 2014

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: Arm 1: EMST The experimental group receives five weeks of expiratory muscle strength training (EMST) using a positive pressure threshold device	Device: EMST; Pressure threshold device (Expiratory Muscle Strength Trainer) targeted at increase muscle force generation of expiratory and submental muscles.; Other Names: EMST 150
Sham Comparator: Arm 2: Sham group The Sham group undergoes the same 5-week EMST exercise as the experimental group using the same device but without a spring for minimal pressure load	Device: Sham; The same device just like the EMST but does not provide a load on the target muscle group

Outcome Measures

Primary Outcome Measures

1. Maximum Expiratory Pressure (MEP) [at baseline and again after 5-week EMST exercise]

   Expiratory pressure generating capacity assessed via handheld manometer.

2. Penetration-Aspiration Scale Score [at baseline and again after 5-week EMST exercise]

   The Penetration-Aspiration Scale (PAS) was used to measure swallow safety. PAS is an 8 point ordinal scale for quantification of penetration and aspiration. PAS measures the depth to which material enters the airway and if the material is expelled following penetration or aspiration. Categorical groupings of PAS scores include "normal to mild" (1-2), "moderate" (3-5) and "severe" (6-8, indicating that material has passed into the lower airway). These PAS scores may be useful in denoting clinically significant changes (e.g. moderate to mild) resulting from treatment or disease progression. The following table reports the percentage of participants (out of the respective total group participants in EMST and Sham) with changed PAS score of 1 point or more (improving or worsening) and without PAS score changes from pre- to post treatment. The data represent an exploratory quantification without statistical analysis.

3. Swallow-related Quality of Life (SWAL-QOL) [at baseline and after 5-week of EMST exercise]

   The SWAL-QOL is a validated and standardized tool that measures burden; symptom status including pharyngeal, oral, and saliva; fear; and mental health subdomains. Responses are determined according to an ordinal scale where 1 equals a severe problem and 5 equals no problem. The SWAL-QOL provides an overall score as well as subscale scores. Subjects rate quality of life as follows (expressed as percentage of the possible perfect score): little to no impact (81% - 100%), mild impact (61% - 80%), moderate impact (41% - 60%), severe impact (21% - 40%), and profound impact (0% - 20%).

Eligibility Criteria

Criteria

• Ages Eligible for Study: 35 Years to 80 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Multiple Sclerosis Participants

• Diagnosis of primary, secondary, or relapsing-remitting MS by a neurologist

• Over 85% of the patient populations that come from the study sites demonstrate relapsing-remitting MS with an average relapse frequency of once every 3 years

Parkinson's Disease Participants

• Hoehn & Yahr, stage II and III as indicated by certified movement disorders neurologist

All Participants

• Between 35 and 80 years of age

• Non-smoking or no smoking within the previous five years

• No history of head and neck cancer, asthma or COPD, untreated hypertension

• Sufficient facial muscle strength so as to achieve and maintain adequate lip closure around a circular mouthpiece

• Cognition within normal limits as determined by the: Mini Mental Status Exam (MMSE; 1975No neurological (other than MS or PD) condition which adversely affects respiratory muscle or gas exchange system

• Reduced MEP's compared to published normative data for age and sex

• Reduced expiratory peak flow rates (6-8 L/s for young to middle age adults and 3.6 L/s for 65 and older) during voluntary cough production for age and sex (Bolser, personal communication; Smith-Hammond & Goldstein, 2006)

• Participant report of symptoms related to swallow impairment

Exclusion Criteria:

• DBS

• COPD

• Asthma

• Smoking or smoking within preceding 5 years

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	North Florida/South Georgia Veterans Health System, Gainesville, FL	Gainesville	Florida	United States	32608

Sponsors and Collaborators

• VA Office of Research and Development
• University of Florida

Investigators

• Principal Investigator: Janis J. Daly, PhD MS, North Florida/South Georgia Veterans Health System, Gainesville, FL

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: VA Office of Research and Development
• ClinicalTrials.gov Identifier: NCT00856518
• Other Study ID Numbers: B6576-R
• First Posted: Mar 5, 2009
• Last Update Posted: Feb 10, 2017
• Last Verified: Dec 1, 2016

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by VA Office of Research and Development: Multiple Sclerosis; Parkinson's disease; Dysphagia
• Additional relevant MeSH terms: Parkinson Disease; Multiple Sclerosis; Neurodegenerative Diseases; Sclerosis

Study Results

Participant Flow

Arm/Group Title	Arm 1: EMST	Arm 2: Sham Group
Arm/Group Description	Experimental Group Expiratory Muscle Strength Trainer: Pressure threshold device targeted at increase muscle force generation of expiratory and submental muscles.	sham group sham device: Looks just like the EMST device but does not provide a load on the target muscle group
Period Title: Overall Study
STARTED	24	18
COMPLETED	20	16
NOT COMPLETED	4	2

Baseline Characteristics

Arm/Group Title	Arm 1: EMST	Arm 2: Sham Group	Total
Arm/Group Description	Expiratory Muscle Strength Trainer: Pressure threshold device targeted at increase muscle force generation of expiratory and submental muscles.	Sham Device: Looks just like the EMST device but does not provide a load on the target muscle group	Total of all reporting groups
Overall Participants	24	18	42
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	53 (9)	58 (8)	55 (9)
Gender (Count of Participants)
Female	18 75%	13 72.2%	31 73.8%
Male	6 25%	5 27.8%	11 26.2%

Outcome Measures

1. Primary Outcome

Title	Maximum Expiratory Pressure (MEP)
Description	Expiratory pressure generating capacity assessed via handheld manometer.
Time Frame	at baseline and again after 5-week EMST exercise

Outcome Measure Data

Analysis Population Description

Out of the 42 recruited patients, 6 withdrew following the baseline MEP testing, citing travel or loss of interest as the reason. Therefore, 36 subjects were reported for the MEP test (n = 20 for EMST, n = 16 for sham).

Arm/Group Title	Arm 1: EMST	Arm 2: Sham
Arm/Group Description	EMST Group Expiratory Muscle Strength Trainer: Pressure threshold device targeted at increase muscle force generation of expiratory and submental muscles.	Sham group sham device: Looks just like the EMST device but does not provide a load on the target muscle group
Measure Participants	20	16
baseline	78.6 (30.72)	75.56 (27.68)
post-training	99.00 (32.97)	99.38 (37.59)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST, Arm 2: Sham
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.899
	Comments	EMST arm vs. Sham arm baseline MEP value comparison
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST, Arm 2: Sham
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.946
	Comments
	Method	Plum Ordinal Regression Test
	Comments	EMST arm vs. Sham arm group comparison of treatment response

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.00042
	Comments
	Method	t-test, 2 sided
	Comments	EMST arm post vs. pre-MEP comparison

Statistical Analysis 4

Statistical Analysis Overview	Comparison Group Selection	Arm 2: Sham
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.0019
	Comments	Sham arm post vs. pre-MEP comparison
	Method	t-test, 2 sided
	Comments

2. Primary Outcome

Title	Penetration-Aspiration Scale Score
Description	The Penetration-Aspiration Scale (PAS) was used to measure swallow safety. PAS is an 8 point ordinal scale for quantification of penetration and aspiration. PAS measures the depth to which material enters the airway and if the material is expelled following penetration or aspiration. Categorical groupings of PAS scores include "normal to mild" (1-2), "moderate" (3-5) and "severe" (6-8, indicating that material has passed into the lower airway). These PAS scores may be useful in denoting clinically significant changes (e.g. moderate to mild) resulting from treatment or disease progression. The following table reports the percentage of participants (out of the respective total group participants in EMST and Sham) with changed PAS score of 1 point or more (improving or worsening) and without PAS score changes from pre- to post treatment. The data represent an exploratory quantification without statistical analysis.
Time Frame	at baseline and again after 5-week EMST exercise

Outcome Measure Data

Analysis Population Description

Out of the 42 recruited patients, 8 failed to complete the PAS test either at baseline or post training testing. Therefore, 34 subjects were reported for the PAS test (n = 20 for EMST, n = 14 for sham).

Arm/Group Title	Arm 1: EMST	Arm 2: Sham
Arm/Group Description	EMST Group Expiratory Muscle Strength Trainer: Pressure threshold device targeted at increase muscle force generation of expiratory and submental muscles.	Sham group sham device: Looks just like the EMST device but does not provide a load on the target muscle group
Measure Participants	20	14
with improved PAS after training	40.0 166.7%	14.3 79.4%
with unchanged PAS after training	45.0 187.5%	64.3 357.2%
with worsened PAS after training	15.0 62.5%	21.4 118.9%

3. Primary Outcome

Title	Swallow-related Quality of Life (SWAL-QOL)
Description	The SWAL-QOL is a validated and standardized tool that measures burden; symptom status including pharyngeal, oral, and saliva; fear; and mental health subdomains. Responses are determined according to an ordinal scale where 1 equals a severe problem and 5 equals no problem. The SWAL-QOL provides an overall score as well as subscale scores. Subjects rate quality of life as follows (expressed as percentage of the possible perfect score): little to no impact (81% - 100%), mild impact (61% - 80%), moderate impact (41% - 60%), severe impact (21% - 40%), and profound impact (0% - 20%).
Time Frame	at baseline and after 5-week of EMST exercise

Outcome Measure Data

Analysis Population Description

Out of the total 42 recruited study participants, ten either did not show up for the SWAL-QOL test or only partially answered the questionnaire. Thus, 32 remaining participants (n = 19 for EMST, and n = 13 for Sham) were reported.

Arm/Group Title	Arm 1: EMST	Arm 2: Sham Group
Arm/Group Description	The experimental group receives five weeks of expiratory muscle strength training (EMST) using a positive pressure threshold device Expiratory Muscle Strength Trainer: Pressure threshold device targeted at increase muscle force generation of expiratory and submental muscles.	The sham group undergoes the same 5-week EMST exercise as the experimental group using the same device but without a spring for minimal pressure load sham device: Looks just like the EMST device but does not provide a load on the target muscle group
Measure Participants	19	13
pre-training total SWAL-QOL	83.3 (11.5)	81.4 (10.9)
post-traing total SWAL-QOL	88.1 (10.8)	84.4 (8.5)
pre-training Burden	90.0 (20.0)	80.8 (25.0)
post-training Burden	97.4 (7.3)	86.2 (18.9)
pre-training Pharyngeal	73.5 (16.9)	75.4 (13.0)
post-training Pharyngeal	82.4 (16.6)	77.6 (11.6)
pre-training Saliva	84.2 (16.2)	83.1 (13.5)
post-training Saliva	92.3 (9.5)	83.6 (15.8)
pre-training Oral	95.3 (7.0)	91.2 (13.1)
post-training Oral	96.9 (4.5)	91.2 (9.8)
pre-training Fear	80.5 (22.2)	79.2 (14.8)
post-training Fear	89.2 (20.4)	84.6 (11.3)
pre-training Mental Health	87.2 (21.5)	83.4 (16.5)
post-training Mental Health	94.1 (18.2)	90.2 (13.0)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST, Arm 2: Sham
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	> 0.05
	Comments	EMST arm vs. Sham arm baseline total score and subscale score comparison
	Method	Wilcoxon (Mann-Whitney)
	Comments

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST, Arm 2: Sham
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	> 0.05
	Comments
	Method	Plum Ordinal Regression Test
	Comments	EMST arm vs. Sham arm group comparison of treatment response for total score and all subscale scores except for Burden and Pharyngeal domains

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST, Arm 2: Sham
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.014
	Comments
	Method	Plum Ordinal Regression Test
	Comments	EMST arm vs. Sham arm group comparison of treatment response in Burden domain

Statistical Analysis 4

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST, Arm 2: Sham
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.022
	Comments
	Method	Plum Ordinal Regression test
	Comments	EMST arm vs. Sham arm group comparison of treatment response in Pharyngeal domain

Statistical Analysis 5

Statistical Analysis Overview	Comparison Group Selection	Arm 2: Sham
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	>0.05
	Comments	Sham arm post vs. pre-treatment comparison for the total SWAL-QOL score and subscale scores except for the Burden and Mental Health domains.
	Method	Wilcoxon Signed Ranks Test
	Comments

Statistical Analysis 6

Statistical Analysis Overview	Comparison Group Selection	Arm 2: Sham
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.038
	Comments
	Method	Wilcoxon Signed Ranks Test
	Comments	Sham arm post vs. pre-treatment comparison in Burden domain

Statistical Analysis 7

Statistical Analysis Overview	Comparison Group Selection	Arm 2: Sham
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.031
	Comments
	Method	Wilcoxon Signed Ranks Test
	Comments	Sham arm post vs. pre-treatment comparison in Mental Health domain

Statistical Analysis 8

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.027
	Comments
	Method	Wilcoxon Signed Ranks Test
	Comments	EMST arm post vs. pre-treatment comparison in Burden domain

Statistical Analysis 9

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.016
	Comments
	Method	Wilcoxon Signed Ranks Test
	Comments	EMST arm post vs. pre-treatment comparison in Mental Health domain

Statistical Analysis 10

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.007
	Comments
	Method	Wilcoxon Signed Ranks Test
	Comments	EMST arm post vs. pre-treatment comparison in Pharyngeal domain

Statistical Analysis 11

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.036
	Comments
	Method	Wilcoxon Signed Ranks Test
	Comments	EMST arm post vs. pre-treatment comparison in Saliva domain

Statistical Analysis 12

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.004
	Comments
	Method	Wilcoxon Signed Ranks Test
	Comments	EMST arm post vs. pre-treatment comparison in Fear domain

Statistical Analysis 13

Statistical Analysis Overview	Comparison Group Selection	Arm 1: EMST
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.016
	Comments
	Method	Wilcoxon Signed Ranks Test
	Comments	EMST arm post vs. pre-treatment comparison in Total SWAL-QOL score

Adverse Events

Time Frame	Seven-week period (baseline testing at week 1, then 5 weeks of the experimental training, and post-training testing at week 7)
Arm/Group Title	Arm 1: EMST		Arm 2: Sham Group
Arm/Group Description	Expiratory Muscle Strength Trainer: Pressure threshold device targeted at increase muscle force generation of expiratory and submental muscles.		Sham Device: Looks just like the EMST device but does not provide a load on the target muscle group
All Cause Mortality
	Arm 1: EMST		Arm 2: Sham Group
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	Arm 1: EMST		Arm 2: Sham Group
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/24 (0%)		0/18 (0%)
Other (Not Including Serious) Adverse Events
	Arm 1: EMST		Arm 2: Sham Group
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/24 (0%)		0/18 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.